High-efficiency wild-type-free AAV helper functions
First Claim
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1. A nucleic acid molecule which encodes one or more AAV helper functions, said nucleic acid molecule comprising:
- an AAV rep coding region;
an AAV cap coding region; and
a nucleotide sequence comprising a modified AAV p5 promoter region, such that the modified AAV p5 promoter region no longer functions in transcription initiation.
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Abstract
The present invention provides methods and compositions for producing high titer, wild-type-free preparations of recombinant AAV ("rAAV") virions. The compositons of the present invention include novel nucleic acids encoding AAV helper functions and AAV helper function vectors. The present invention also includes host cells transfected by the claimed nucleic acids, methods of using the claimed vectors, and rAAV virions produced by such methods.
74 Citations
25 Claims
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1. A nucleic acid molecule which encodes one or more AAV helper functions, said nucleic acid molecule comprising:
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an AAV rep coding region; an AAV cap coding region; and a nucleotide sequence comprising a modified AAV p5 promoter region, such that the modified AAV p5 promoter region no longer functions in transcription initiation. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8)
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9. A nucleic acid molecule which encodes one or more AAV helper functions, said nucleic acid molecule comprising:
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an AAV rep coding region; an AAV cap coding region; and a nucleotide sequence comprising a modified AAV p5 promoter region, wherein the modified p5 promoter region lacks an intact TATA box. - View Dependent Claims (10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25)
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Specification